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van CCT (UK)

van CCT (UK)

Efficacy of genestein as food supplement in patients with the Sanfilippo syndrome (an extension study).

- candidate number8201
- NTR NumberNTR2402
- ISRCTNISRCTN wordt niet meer aangevraagd.
- Date ISRCTN created
- date ISRCTN requested
- Date Registered NTR5-jul-2010
- Secondary IDs10/080 MEC AMC
- Public TitleEfficacy of genestein as food supplement in patients with the Sanfilippo syndrome (an extension study).
- Scientific TitleEfficacy of genestein as food supplement in lpatienst with the Sanfilippo syndrome (an extension study).
- hypothesisGenestein inhibits the synthesis of heparan sulphate and can possibly slow down disease progression in Sanfilippo Syndrome patients.
- Healt Condition(s) or Problem(s) studiedSanfilippo syndrome (MPS III)
- Inclusion criteriaPatients participated in the initial double blind placebo controlled study on the effect of genestein in a cross-over design.
- Exclusion criteriaThe parent or legal representative is unwilling to participate.
- mec approval receivedyes
- multicenter trialno
- randomisedno
- groupParallel
- TypeSingle arm
- Studytypeintervention
- planned startdate 12-jul-2010
- planned closingdate1-aug-2011
- Target number of participants28
- InterventionsDuring a 12 month period all particpating patients will receive genestein as food supplement. Genestein dose will be calculated according to the weight of the patients (10 mg/kg/day). The start assessment of this study equals the last assessment of the double blind controlled trial.
- Primary outcomeDoes genestein significantly decrease urinary GAGs (in particular heparan sulphate) excretion in Sanfilippo patients?
- Secondary outcome1. Does genestein decrease serum heparan sulphate concentration?
2. Does genestein decrease accumulation of heparan sulphate in skin biopsies?
3. Does hair morphology improve in MPS III patients using genestein?
4. What is the effect of genestein on behaviour?
5. Does genestein improve neurocognitive outcome?
- TimepointsBaseline, 6 months and 12 months.
- Trial web siteN/A
- statusinclusion stopped: follow-up
- Sponsor/Initiator Academic Medical Center (AMC), Amsterdam
- Funding
(Source(s) of Monetary or Material Support)
Stichting Kinderen en Kansen
- PublicationsN/A
- Brief summarySanfilippo syndrome (MPS III) is an inborn errors of lysosomal degradation of the glycosaminoglycan heparan sulphate (GAGs). Accumulation of heparan sulfate, results in progressive and severe mental deterioration, which is the clinical hallmark of MPS III. Genestein (an isoflavone) is a natural component of the soy-bean. Genistein inhibits GAGs synthesis in vitro and a recent small and open-label study suggested clinical relevant effects of genestein in MPS III patients. In June 2009 a double blind placebo controlled study on the effect of genestein was initiated in the Netherlands. Thirty patients with MPS III type A, B or C are included in this study and receiving either placebo or genestein (10 mg/kg) during two 6 month periods in a cross-over design The outcome of genestein on urinary GAG excretion, plasma heparan sulphate, tissue (skin biopsy) concentration of heparan sulphate, hair structure, cognitive state and behavioural abnormalities will be studied. Currently the trial is still ongoing and first results are expected the end of 2010. Our study will be the extension study of the previous mentioned study.
- Main changes (audit trail)
- RECORD5-jul-2010 - 27-jul-2010

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